Patient Perspectives on the Value of Patient Preference Information in Regulatory Decision Making

Background: There is increasing interest in involving patient preferences for benefits and risks in regulatory decision making. Therefore, it is essential to identify patient perspectives regarding the value of patient preference information (PPI). Objectives: The aim of this study was to explore how patients with rheumatoid arthritis (RA) value the use of PPI in regulatory decision making regarding medical products. Methods: Regulators and patients with RA were interviewed to gather initial insights into opinions on the use of PPI in regulatory decisions regarding medical products. The interviews were used to draft and validate the interview guide for focus groups with patients with RA. Participants were purposively sampled in collaboration with the Swedish Rheumatism Association in Stockholm and Uppsala. Each focus group consisted of three to six patients (18 in total). All interviews were audio-recorded, transcribed verbatim, and analysed using content analysis. Results: According to the participants, PPI could lead to regulators considering patients’ needs, lifestyles and well-being when making decisions. PPI was important in all stages of the medical product lifecycle. Participants reported that, when participating in a preference study, it is important to be well-informed about the use of the study and the development, components, administration, and risks related to the medical products. Conclusions: Patients thought PPI could be valuable to consider in regulatory decisions. It is essential for patients to be well-informed when asked for their preferences. Research on information materials to inform patients in preference studies is needed to increase the value of PPI in regulatory decision making

Patients accept therapy using embryonic stem cells for Parkinson’s disease:a discrete choice experiment

Background: New disease-modifying ways to treat Parkinson’s disease (PD) may soon become a reality with intracerebral transplantation of cell products produced from human embryonic stem cells (hESCs). The aim of this study was to assess what factors influence preferences of patients with PD regarding stem-cell based therapies to treat PD in the future. Methods: Patients with PD were invited to complete a web-based discrete choice experiment to assess the importance of the following attributes: (i) type of treatment, (ii) aim of treatment, (iii) available knowledge of the different types of treatments, (iv) effect on symptoms, and (v) risk for severe side effects. Latent class conditional logistic regression models were used to determine preference estimates and heterogeneity in respondents’ preferences. Results: A substantial difference in respondents’ preferences was observed in three latent preference patterns (classes). “Effect on symptoms” was the most important attribute in class 1, closely followed by “type of treatment,” with medications as preferred to other treatment alternatives. Effect on symptoms was also the most important attribute in class 2, with treatment with hESCs preferred over other treatment alternatives. Likewise for class 3, that mainly focused on “type of treatment” in the decision-making. Respondents’ class membership was influenced by their experience in treatment, side effects, and advanced treatment therapy as well as religious beliefs. Conclusions: Most of the respondents would accept a treatment with products emanating from hESCs, regardless of views on the moral status of embryos. Preferences of patients with PD may provide guidance in clinical decision-making regarding treatments deriving from stem cells.</p

How can patient preferences be used and communicated in the regulatory evaluation of medicinal products? Findings and recommendations from IMI PREFER and call to action

Objective: Patients have unique insights and are (in-)directly affected by each decision taken throughout the life cycle of medicinal products. Patient preference studies (PPS) assess what matters most to patients, how much, and what trade-offs patients are willing to make. IMI PREFER was a six-year European public-private partnership under the Innovative Medicines Initiative that developed recommendations on how to assess and use PPS in medical product decision-making, including in the regulatory evaluation of medicinal products. This paper aims to summarize findings and recommendations from IMI PREFER regarding i) PPS applications in regulatory evaluation, ii) when and how to consult with regulators on PPS, iii) how to reflect PPS in regulatory communication and iv) barriers and open questions for PPS in regulatory decision-making.Methods: PREFER performed six literature reviews, 143 interviews and eight focus group discussions with regulators, patient representatives, industry representatives, Health Technology Assessment bodies, payers, academics, and clincians between October 2016 and May 2022.Results: i) With respect to PPS applications, prior to the conduct of clinical trials of medicinal products, PPS could inform regulators’ understanding of patients’ unmet needs and relevant endpoints during horizon scanning activities and scientific advice. During the evaluation of a marketing authorization application, PPS could inform: a) the assessment of whether a product meets an unmet need, b) whether patient-relevant clinical trial endpoints and outcomes were studied, c) the understanding of patient-relevant effect sizes and acceptable trade-offs, and d) the identification of key (un-)favorable effects and uncertainties. ii) With respect to consulting with regulators on PPS, PPS researchers should ideally have early discussions with regulators (e.g., during scientific advice) on the PPS design and research questions. iii) Regarding external PPS communication, PPS could be reflected in the assessment report and product information (e.g., the European Public Assessment Report and the Summary of Product Characteristics). iv) Barriers relevant to the use of PPS in regulatory evaluation include a lack of PPS use cases and demonstrated impact on regulatory decision-making, and need for (financial) incentives, guidance and quality criteria for implementing PPS results in regulatory decision-making. Open questions concerning regulatory PPS use include: a) should a product independent broad approach to the design of PPS be taken and/or a product-specific one, b) who should optimally be financing, designing, conducting, and coordinating PPS, c) when (within and/or outside clinical trials) to perform PPS, and d) how can PPS use best be operationalized in regulatory decisions.Conclusion: PPS have high potential to inform regulators on key unmet needs, endpoints, benefits, and risks that matter most to patients and their acceptable trade-offs. Regulatory guidelines, templates and checklists, together with incentives are needed to foster structural and transparent PPS submission and evaluation in regulatory decision-making. More PPS case studies should be conducted and submitted for regulatory assessment to enable regulatory discussion and increase regulators’ experience with PPS implementation and communication in regulatory evaluations

Getting a Say : Bringing patients’ views on benefit-risk into medical approvals

The focus of this thesis is a new quantitative approach to consider patient preferences on benefits and risks in medical approvals. The overall aim of this thesis was to explore how patient preference information may be relevant to regulatory marketing authorisation decisions. Study I provides an overview of the different decision-processes of industry, regulatory agencies and health technology assessment bodies/reimbursement agencies along the medical product lifecycle. In total, 15 decision points with the potential to include patient preference information were identified.  Study II was an exploration of the patient perspective regarding the use of patient preference information in regulatory marketing authorisation decisions. Patients emphasised the need to have a say in decisions affecting their health and to be properly informed about potential risks and benefits of medical products.  Study III assessed patient preferences on benefits and risks of Rheumatoid Arthritis treatments. Results revealed that patients’ preferences differed substantially. The three most important treatment attributes for patients with rheumatoid arthritis were: the probability of severe side effects, treatment effectiveness and route of administration. Those placing relatively more importance on treatment effectiveness were willing to acceptance higher risk levels of side effects.  Study IV aimed to determine the influence of an educational tool, compared with traditional written information on patient preferences. It was found that those respondents receiving the educational tool focused more on the potential side effects than those receiving written information.  Patient preference information has the potential to reveal patients’ preferences on benefits and risks with scientific rigour and can therefore be weighed against clinical data. This thesis supports the development of a structured approach to learn about patient preferences on benefits and risks in medical approval

Miljömanagement och uppföljning av dess ekonomiska och miljömässiga prestanda : En studie inom svensk detaljhandel

Company managers have increasingly adopted environmental management in the 2000’s, and the relationship between environmental management and financial performance has been a subject for discussion during the last decades because of inconsistent results. The aim of this study is to investigate this relationship between environmental management and financial performance within companies, and to analyse how Swedish retail companies are evaluating the environmental and financial results from environmental management. The methodology used for the study is a literature review to capture the current knowledge area in research, a study of companies’ sustainability reports and an interview study to collect empirical evidence from the retail sector. The literature study shows a positive result between environmental performance and financial performance, a positive relationship between environmental management and financial performance, and non-conclusive data of the relationship between environmental management systems and financial performance. Furthermore it shows that companies are measuring and reporting environmental performance but no or few companies are reporting the financial outcome from environmental management, however companies seem to measure cost savings from environmental management. From this study, no consensus of a general methodology of the measuring of the financial outcomes from environmental management has been found. The findings are not representing the entire retail sector and should rather be seen as empirical evidence from a number of retail companies. The conclusion made was that the ambitions within environmental management differ within one industry sector, and that there seem to be no general methodology for measuring the financial outcome from environmental management.Miljömanagement har införts av fler och fler företagsledare under 2000-talet, och kopplingen mellan miljömanagement och affärsnytta har varit ett ämne för debatt under de senaste decennierna då resultaten ofta varit motsägande. Syftet med den här studien är att undersöka relationen mellan miljömanagement och affärsnytta inom företag, samt att analysera hur svensk detaljhandel utvärderar och mäter de miljömässiga och ekonomiska resultaten från miljömanagement. Metoden som har använts är en litteraturstudie för att fånga det nuvarande kunskapsläget i forskningen, en studie av företags hållbarhetsrapporter samt en intervjustudie för att samla empirisk data från detaljhandeln. Litteraturstudien visar att relationen mellan miljöprestanda och affärsnytta är positiv, att relationen mellan miljömanagement och affärsnytta är positiv samt en icke-bestämd relation mellan miljöledningssystem och affärsnytta. Studien visar även att företag inom detaljhandeln mäter och rapporterar miljöprestanda men inga eller få företag rapporterar affärsnytta från miljömanagement, däremot förekommer mätningar av kostnadsbesparingar hos flera företag. Inga generella metoder för att mäta affärsnyttan av miljömanagement har identifierats i studien. Resultatet i den här studien representerar inte hela detaljhandeln, utan är en samling av empiriska data från ett urval av företag. Slutsatsen från studien är dels att ambitionen inom miljömanagement skiljer sig åt inom olika företag inom detaljhandeln, samt att det inte verkar finnas någon generell metod för att mäta affärsnyttan av miljömanagement

Getting a Say : Bringing patients’ views on benefit-risk into medical approvals

The focus of this thesis is a new quantitative approach to consider patient preferences on benefits and risks in medical approvals. The overall aim of this thesis was to explore how patient preference information may be relevant to regulatory marketing authorisation decisions. Study I provides an overview of the different decision-processes of industry, regulatory agencies and health technology assessment bodies/reimbursement agencies along the medical product lifecycle. In total, 15 decision points with the potential to include patient preference information were identified.  Study II was an exploration of the patient perspective regarding the use of patient preference information in regulatory marketing authorisation decisions. Patients emphasised the need to have a say in decisions affecting their health and to be properly informed about potential risks and benefits of medical products.  Study III assessed patient preferences on benefits and risks of Rheumatoid Arthritis treatments. Results revealed that patients’ preferences differed substantially. The three most important treatment attributes for patients with rheumatoid arthritis were: the probability of severe side effects, treatment effectiveness and route of administration. Those placing relatively more importance on treatment effectiveness were willing to acceptance higher risk levels of side effects.  Study IV aimed to determine the influence of an educational tool, compared with traditional written information on patient preferences. It was found that those respondents receiving the educational tool focused more on the potential side effects than those receiving written information.  Patient preference information has the potential to reveal patients’ preferences on benefits and risks with scientific rigour and can therefore be weighed against clinical data. This thesis supports the development of a structured approach to learn about patient preferences on benefits and risks in medical approval

Conversion to a Circular Industry : Success and limiting factors to convert Högdalen Industrial Area into an eco-industrial park

The concept of eco-industrial parks is a way to minimize environmental impact and resource use from an industrial area, and in this study the concept is applied to Högdalen industrial area in Stockholm. An interview study has been conducted with key actors in the area, which found for instance that there were a good cooperation will among the actors and some material and energy exchanges occurring. This was then compared to a literature study of the limiting and success factors of the development of an eco-industrial parks. A quarter of the identified factors were determined to have good prospects, a fifth was partly fulfilled, a fifth had bad prospects, a fifth was unknown and 15 % was unknown but relevant to uncover in this stage of development.  For the future, it is suggested that: more interviews should be done with more actors in the area, Cleantech Högdalen should change focus to develop an eco-industrial park, more energy and material flows should be mapped and to gather information of unknown and relevant factors. The conclusion of this study is that there is potential to develop Högdalen Industrial Area into an eco-industrial park but there are some knowledge gaps about the quantitative minimization of resources and environmental impact. Konceptet eco-industrial parks bygger på att minimera klimatpåverkan och resursanvändning från ett industriområde, i den här studien är konceptet applicerat på Högdalens industriområde i Stockholm. En intervjustudie har utförts i området, där nyckelaktörer har intervjuats, studien visade bland annat att det är en generellt sätt god samarbetsvilja i området samt att några energi-och materialutbyten sker. Intervjustudien har jämförts med en literaturstudie av framgångsfaktorer och begränsande faktorer för utvecklingen av en eco-industrial park. En fjärdedel av de identifierade faktorerna har goda utsikter, en femtedel är delvis uppfyllda, en femtedel har dåliga utsikter, en femtedel är okända och 15 % är okända men relevanta i detta steg av utvecklingen. För framtiden föreslås intervjustudier med fler aktörer i området, Cleantech Högdalen bör ändra sitt fokus till att utveckla en eco-industrial park i området, fler energi- och materialutbyten bör kartläggas samt att information bör samlas om de faktorer som är okända och relevanta. Slutsatsen av studien är att det finns en potential att utveckla Högdalens industriområde till en eco-industrial park men att det är kunskapsluckor kring den kvantitativa potentialen att minimera resursanvändning och således också klimatpåverkan.

Experiences of individuals with rheumatoid arthritis interacting with health care and the use of a digital self-care application: a qualitative interview study

Objectives Over the last few decades, there have been significant improvements in the treatment of rheumatoid arthritis (RA), with the development of new treatments and guidelines for teamwork and patient self-care and access to digital tools. This study aimed to explore the experiences of individuals with RA interacting with healthcare. It also looked at how a self-care application, an educational programme called the ‘healthcare encounter’, improved patient–doctor communication.Design Semistructured interviews were conducted, and qualitative content analysis was performed.Setting The potential participants, individuals with established, or under investigation for, RA diagnosis at rheumatology clinics in Sweden, were asked to participate in the study via a digital self-care application called the Elsa Science Self-care app.Participants Ten interviews were performed with participants from nine clinics following a meeting with the rheumatologist or other healthcare personnel between September 2022 and October 2022. Phrases, sentences or paragraphs referring to experiences from healthcare meetings and opinions about the digital programme were identified and coded. Codes that reflected similar concepts were grouped; subcategories were formulated, and categories were connected to their experiences and opinions.Results Among our participants, three main categories emerged: the availability of healthcare, individual efforts to have a healthier life and personal interaction with healthcare. Participants described that the ‘healthcare encounter’ educational programme can be a source of information, which confirms, supports and creates a sense of control.Conclusion The participants valued being seen and taking part in a dialogue when they had prepared themselves (observed symptoms over time and prepared questions). The implementation of digital self-care applications might need to be incorporated into the healthcare setting, so that both the patients and the healthcare personnel have a shared understanding. Collaboration is essential in this context

Would you consider donating your left-over embryos to treat Parkinson’s disease? Interviews with individuals that underwent IVF in Sweden

Background: Parkinson’s disease (PD) has been considered to be one of the most promising target diseases for forthcoming cell-based therapy. The aim of this study is to explore the views of individuals with cryopreserved embryos on using human embryonic stem cells for treating PD. Methods: The study was performed as a qualitative, semi-structured interview study in June–October 2020. Participants were recruited at a private fertility clinic located in one of the larger Swedish cities. The clinic provides both publicly financed and privately financed IVF-treatments. All interviews were performed by telephone and analyzed using thematic content analysis. Five main categories emerged from 27 sub-categories. Results: In total, 18 interviews were performed with 22 individuals, as either a couple (n = 16) or separately (n = 6). Participants had different views on what a cryopreserved embryo is. Some participants addressed cryopreserved embryos as ‘a lump of cells’, and some in terms of their ‘unborn child’. Conditions for donation of cryopreserved embryos for cell-based treatment in PD were: not losing control of what is happening to the embryo, that donating must be voluntary and based on informed consent with time for reflection, reimbursement, equality and transparency. Conclusions: Using cryopreserved embryos to treat PD is associated with fundamental ethical and practical issues. This study shows that IVF couples with left-over embryos may be supportive but there is a need for future research to assess people’s views on using cryopreserved embryos for cell-based treatment in PD on a more aggregated level

Does being exposed to an educational tool influence patient preferences?:The influence of an educational tool on patient preferences assessed by a discrete choice experiment.

Objectives: There is an increased interest in patient preferences informing the development and authorisation of medical products. A requirement for robust and meaningful results of such studies is that patients adequately understand the risks and benefits associated with treatments for which their preferences are elicited. This study aims to determine the influence of an educational tool, compared with traditional written information on patient preferences elicited in a discrete choice experiment (DCE). Methods: Treatment preferences of Swedish patients with rheumatoid arthritis (RA) were assessed using a DCE. Patients were recruited via clinics, a research panel, and the Swedish Rheumatism Association. Respondents received training materials either as plain written text or as an online educational tool. The educational tool was designed to enhance understanding of the written text by using graphics, pictograms, icon arrays, spoken text, and click-on functions. Data were analysed using random parameter logit models. Results: 675 patients with RA were included in the analysis. The patients received either a written information (n = 358) or information via an educational tool (n = 317). Respondents receiving the educational tool placed relatively more importance on all included side effects in their decision making, compared to respondents receiving the written text, who placed greater importance on treatment effectiveness and administration methods. Conclusion: Compared to the respondents receiving the written text, the decisions of respondents receiving the educational tool were more influenced by medication side effects. Further research is needed to provide guidance on how and when to use educational tools to inform and elicit patients’ preferences. Practice implications: The ways in which attributes are presented to patients significantly impacts preferences measured in a DCE